Navigation Links
Promising new drug targets identified for Huntington's disease

Research funded by the Wellcome Trust has provided a number of promising new drug targets for Huntington's disease, a neurodegenerative disease. Scientists at the University of Cambridge have identified a number of candidate drugs to investigate further which encourage cells to "eat" the malformed proteins that lead to the disease.

Huntington's disease is one of a number of degenerative diseases marked by build up of a malformed proteins in brain cells, mainly in the basal ganglia and the cerebral cortex. Normally, cells dispose of or recycle their waste material, including unwanted or misfolded proteins, through a process known as autophagy, or 'self-eating'.

The group of Professor David Rubinsztein, a Wellcome Trust Senior Clinical Fellow at the University of Cambridge, has previously shown that stimulating autophagy in the cells can be an effective way of preventing the malformed proteins from building up. However, there are currently no treatments available that slow the neurodegeneration in people with Huntington's disease. Rapamycin, an immunosuppressant used to lower the body's natural immunity in patients who receive kidney transplants, is the most promising candidate drug currently available but can have significant side effects.

Now, in research published today online in the journal Nature Chemical Biology, Professor Rubinsztein and colleagues have shown that a number of FDA-approved drugs for treatments such as migraine and hypertension are able to stimulate autophagy in fruit flies and zebrafish through unexpected pathways.

"By screening a number of drugs that have already been shown to be safe in humans, we have been able to identify some unexpected and very promising pathways involved in Huntington's," says Professor Rubinsztein. "In collaboration with Cahir OKanes group in Cambridge and Summit Plc, we have shown that these drugs can alleviate the toxicity of the Huntingtons disease mutation in cell-based, fly and zebrafish models. The big question for us is whether they will do the same in humans."

One of the drugs tested, verapamil, which is currently used to treat high blood pressure and heart arrhythmias (among other indications), inhibits the influx of calcium into cells which, in turn, appears to regulate autophagy. Similarly, clonidine, currently used to treat hypertension or migraine, appears to work on autophagy by decreasing levels of cAMP, a molecule that is important in many biological processes.

If the drugs can stimulate autophagy effectively over long-term periods in human brains, then they may have the potential to help delay the onset of Huntington's disease. The candidate drugs are relatively safe and well tolerated when used to treat the diseases they were designed for. A minimal side-effect profile would be highly desirable for a drug treatment aiming to delay the onset or slow the progress of Huntington's. Such drugs may need to be taken for decades, and even moderate side effects may discourage people from taking them over a long period.

"We know the genetics of Huntington's disease and can predict the majority of people at risk," says Professor Rubinsztein. "If we can find a safe, well tolerated drug, then a person at risk could be placed on a drug regime to help prevent onset. It is much easier to stop something happening than having to treat it once it has started."

Professor Rubinsztein and colleagues will shortly begin testing the drugs in other animal models to evaluate their safety and efficacy.


Contact: Craig Brierley
Wellcome Trust

Related biology news :

1. UIC researchers find promising new targets for antibiotics
2. New book defines promising young field of adult neurogenesis
3. Sirtris unveils promising, novel SIRT1 activators for treating diseases of aging
4. Cancer and arthritis therapy may be promising treatment for diabetes
5. Unique whey protein is promising supplement for strict PKU diet
6. Nicotinic receptors may be important targets for treatment of multiple addictions
7. Central targets may hinder wider waste management objectives
8. New X-ray technique targets terrorists and tumors
9. New drug targets may fight tuberculosis and other bacterial infections in novel way
10. Study of successful drug targets could hasten development of new medications
11. A screening strategy using zebrafish targets genes that protect against hearing loss
Post Your Comments:
(Date:10/29/2015)... BOSTON , Oct. 29, 2015  Connected health ... phenomena driving the explosion of technology-enabled health and wellness, ... his new book, The Internet of Healthy ... apps, sensors or smartphones even existed, Dr. Kvedar, vice ... model of health care delivery, moving care from the ...
(Date:10/29/2015)... NEW YORK , Oct. 29, 2015 /PRNewswire/ ... wearable technology, announced a partnership with 2XU, a ... accessories, to deliver a smart hat with advanced ... runners and other athletes to monitor key biometrics ... of the strategic partnership, the two companies will bring ...
(Date:10/27/2015)... , October 27, 2015 ... Automated Semantic Gaze Mapping technology (ASGM) automatically maps data ... Eye Tracking Glasses , so that they can ... --> Munich, Germany , October ... automatically maps data from mobile eye tracking videos created ...
Breaking Biology News(10 mins):
(Date:11/24/2015)... ... 24, 2015 , ... The United States Golf Association (USGA) today announced Dr. ... Award. Presented annually since 1961, the USGA Green Section Award recognizes an individual’s distinguished ... , Clarke, of Iselin, N.J., is an extension specialist of turfgrass pathology in ...
(Date:11/24/2015)... Therapeutics, Inc. (NASDAQ: HALO ) will be presenting at the ... on Wednesday, December 2 at 9:30 a.m. ET/6:30 a.m. PT . ... a corporate overview. th Annual Oppenheimer Healthcare Conference ... a.m. PT . Jim Mazzola , vice president of ... --> th Annual Oppenheimer Healthcare Conference in ...
(Date:11/24/2015)... Mass. (PRWEB) , ... November 24, 2015 , ... ... to maintain healthy metabolism. But unless it is bound to proteins, copper is ... of Health (NIH), researchers at Worcester Polytechnic Institute (WPI) will conduct a systematic ...
(Date:11/24/2015)... - Aeterna Zentaris Inc. (NASDAQ:  AEZS) (TSX: AEZ) (the "Company"), ... Toronto Stock Exchange, confirms that as of the date ... that would cause the recent movements in the Company,s ... About Aeterna Zentaris Inc. . ... Zentaris is a specialty biopharmaceutical company engaged in developing ...
Breaking Biology Technology: